Longevity, life expectancy, premature mortality: Are they lions and tigers and bears?

Before I set up the context for this post, I am going to throw this out to you. Think of putting rocks on a scale to achieve the weight of, let's say, 1,000 lb. And let's say that you are required to use rocks of similar size. You have a bunch of bricks -- these are your biggest "rocks," and you have a bunch of pebbles like the ones I have in my driveway (perhaps you have them in yours too). In order to get to 1,000 lb, will you need more bricks or more pebbles? I am not trying to trick you. This is just an illustration of the fact that you can get to the same magnitude of a variable (in this case weight) by either using a smaller number of more weighty components (bricks) or a larger number of lighter ones (pebbles). Keep this in mind as you read on below.

I got an interesting comment from Brad F. on my post from yesterday regarding the 10% number for the premature death avoidance attributed to access to medical care. He pointed me to a blog post on the always-informative The Incidental Economist web site which called this a "zombie statistic." Despite having a fifteen-year-old who is an avid fan of zombie fiction and film, I was not familiar with this term, but inferred its meaning pretty easily.

The gist was that when people started to look for the origins of this number, the evidence was difficult to find, and, when discovered, was at best shaky:

Thus, as Austin and Adrianna had found, the 35-year old CDC paper seems to be at the root of the often-cited 10% number; it’s “paper 0,” if you will. But those that continue to reference 10% as an estimate for health care’s contribution to health should know that the only evidence they are referencing is a survey of 40 people, done when Jimmy Carter was president. It’s not evidence-based except by the weakest notions of “evidence.” It’s really a zombie statistic.

My obvious next question was whether a more trustworthy estimate existed for the medical care's contribution to life extension in the US. In my search for a better estimate, I continued to go down the rabbit hole of links, arriving here, the AcademyHealth Blog, landing on the article called "Half of longevity gains due to health care." It was a summary of the attempt by the authors of The Incidental Economist to answer this very question. And what did they find? First, they quoted a NEJM citation from 2006, where it was claimed that 90% of the increases in life expectancy since the 1960s was due to reduction in cardiovascular and neonatal deaths. After meandering through several other sources, the authors concluded that we can attribute about 50% of the responsibility for extending our life to medical interventions.

And that's when I really confused myself. I started thinking about whether premature death and longevity are related, and how they may be related, and are we even talking about the same thing when we invoke each of them.

Premature mortality can be quantified in several ways -- 1). percentage of all deaths that are considered premature, or 2). proportion of people in a population whose death is considered premature (that would be so many cases per 100,000 population). Longevity, on the other hand, is a measure of the average life span of a population. The current life expectancy in the US is 78.8 years. This begs the question of how these two, premature deaths and life expectancy, are numerically related to each other. And can the latter go up without the former going down?

Well, if the language here is consistent with how we speak it, "premature" implies that we know what "timely" means. The definition of a "timely" death must be based on the average life expectancy in a population. This number varies according to certain characteristics of a population, of course, so different subgroups would have a different life expectancy. For example, at any given age, the life expectancy of a person with heart disease should be lower than that for a person who is perfectly healthy. If we can reduce the risk of a premature death in people with heart disease, their life expectancy should edge closer to that of a healthy individual. And, in fact, according to the literature, this has happened in cardiovascular patients, partly due to better treatment of blood pressure, and partly due to fewer people smoking and other healthful lifestyle modifications.

So, it's clear that when death due to a disease is postponed, longevity increases and, ergo, premature deaths drop. It's a bit circular, I know. But here is one interesting detail to consider. Longevity or life expectancy (I use them interchangeably) is an age average. So here is one question: Does the impact on the magnitude of life expectancy gains vary with the age of the population in which premature deaths are avoided? I know, its a clunky question. What I mean is, would you expect life expectancy to go up more, less or same amount if we manage to reduce premature deaths in infancy versus old age? If you consider that life expectancy is an average, then infant mortality attenuates this average severely (think adding a whole bunch of numbers into the denominator without contributing anything to the numerator). So you can imagine, if infant mortality goes down a lot (a big reduction in premature deaths), overall population life expectancy spikes decisively. Reducing premature deaths among the elderly, clearly, by this same calculation, will not result in nearly the same increase in life expectancy.

Another way of looking at this is to consider that a much larger reduction in premature deaths among the elderly (think driveway pebbles) than among infants (those sizable bricks) would be needed in order to reach a similar degree of longevity improvement. A less intuitive corollary of this is that we indeed can have an increase in premature mortality in a group that contributes little to longevity (the elderly) and still witness a large bump in life expectancy with a much smaller reduction in premature deaths within a group with an outsized contribution to longevity (infants). So that answers the second question I posed about these measures -- they can diverge.

Now, on to the estimated contribution of medical care to either or both of these. We have, in fact, witnessed a dramatic reduction in infant mortality. I found this report from Health Resources and Services Administration that infant mortality has dropped from 55.7 per 1,000 live births in 1935 to 6.8 per 1,000 live births in 2007. And here is what the authors cite as reasons:

...dramatic declines in infant mortality rates over the long term were due to large declines in mortality from pneumonia and influenza, birth defects, prematurity and low birthweight, respiratory distress syndrome (RDS), sudden infant death syndrome (SIDS), and injuries. Improvements in living conditions, advances in neonatal medicine and infant heath care, reductions in smoking during pregnancy, and increased access to and use of prenatal care have been suggested as factors responsible for decreases in infant mortality over the past several decades...  

 

And here is an interesting detail: the pace of this drop was a dizzying 3.1% per year on average between 1935 and 2000. However, between 2000 and 2007, the rate went down only from 6.9 to 6.8 per 1,000 live births, a staggering deceleration in this steep decline. A further detail indicates that "much of the statistically significant decline [occurred] in the neonatal period." The implication of this is that the latest declines are due to technology use, most likely among the very premature infants upon delivery. This is the very definition of access to medical care, and falls completely outside of the domain of public health.

Just one more random thought. Reductions in infant and cardiovascular mortality, each a product of both medical and public health interventions, are one side of the life expectancy equation. The other, darker, side is the fact that in some groups and locations in the US, the overall longevity is waning. Much of this phenomenon can be attributed to poverty, environmental factors and poor health behaviors, or, in sum, a reflection of our dismal investment in public health. And, sure, there is a component of access here too. And what about this calculus: Between 1990 and 2010, mortality from cardiovascular disease dropped by about 150,000 per year. That would be an awesome contribution to increased longevity and reduced premature deaths, if it weren't offset by all the deaths (presumably premature) related to the healthcare system itself.

I know that none of this gets to the crux of the matter: What is a reliable estimate of what proportion of the increases in life expectancy can be attributed to access to medical care? But what it does make me appreciate is the complexity of each and every term, every definition, every estimate that we confront daily. This devil, as always, is in the details.


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Infographics: devil in details

I got an e-mail yesterday asking me if I would be interested in displaying an info graphic on my blog. After a few back and forths, I decided to do it. After all, a picture is worth a thousand words. But as with everything, buyer beware: the devil is in the details.

A couple of caveats:
1. I cannot back up all of the numbers myself, and the references at the bottom seem to represent single source data, rather than the totality of the evidence. So take with a grain of salt.
2. There are a couple of notable errors in the graphics:
a. The graphic on other first world nations' spending says that the US spends 2x what they do in Japan. What the graph shows is that this is the case as the proportion of the GDP (also the x-axis is not labeled as such). So, the statement is not entirely accurate.
b. In hospitals overcharging, the caption states that hospitals charge 200% more for meds compared to ex US. The number adds up to 100% more, which is 2x.
3. In general, there are actually some valid reasons why we pay more for stuff in the US. I do not, for example, know if the international data are adjusted for various economic factors, such as purchasing power.
4. The statement that doctors are overpaid is a laugh -- take it from someone who has been in the trenches. I worked dawn till dusk and beyond, nearly every day of every week. I was making barely enough to keep food on the table and a roof over our heads. No lavish vacations, no BMWs, etc. It is not the MDs that are overpaid. Go to the C-suites and corporations, and then you are getting warmer.

Despite the disagreements that I have with the data, I thought it might be a good point of discussion. Would love to hear your thoughts about it.


Created by: Medical Billing and Coding Certification
 
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Why the PPACA hearing is the very definition of insanity

I haven't said much about the SCOTUS hearing of the PPACA, but it is time to break my silence. I have been listening to some of the details of the arguments, and I cannot help but be nauseated. It feels to me like the Justices are behaving like my teen: they take literalness to its absurd limit. Health insurance and cell phones, really? This betrays a complete disregard for probability. What are the chances that you will within your lifetime in the US need to dial 911 in the absence of a landline or another human with a cell phone within shouting distance? And what are the odds that you will at some point in your life require a medical consultation? I rest my case.

The more I think about it, the more convinced I am that the bill should have introduced single payer a priori -- funding our access to medicine through a tax. Yes, a tax. Perhaps the government is not the most efficient agent of this, and the overhaul could have been accomplished through some public-private hybrid model. In the end, as I have said here, our priorities are misaligned, as are our perceptions of what is important in this debate. We spend 97% of all the healthcare money on medicine, and we spend well over 97% of our national discussion about health on access to healthcare and medical interventions, which can only make a 10% difference in our health. The real money, so-to-speak, is in public health, which contributes 60% to our true health and gets only 3% of the expenditures and practically no conversational energy.

So, once again I find myself turning to the wisdom of Albert Einstein, who defined insanity as "doing the same thing over and over again, and expecting different results." The SCOTUS circus is the poster child for this insanity. Whatever the outcome, and I am not at all optimistic about the individual mandate, my sense is that nothing will change until we start paying attention to the root causes of our collective illness.

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How our healthcare spending is like that drunk joke

You know that joke about the drunk crawling around under a street light? A cop comes up to him and asks what he is doing. The drunk explains that he is looking for his wallet. The cop, getting ready to help the man, asks where exactly he dropped it. The drunk points to a distant corner of the dark side of the street. The cop, baffled, inquires why the man is looking here. With inimitable logic the drunk responds, "This is where the light is."

What does this story have to do with anything? Well, I went to a great HIT tweet-up in Cambridge yesterday, organized by Scratch Marketing and led by Janice McCallum. No, they did not at all remind me of the drunk in the joke. But the lively discussion about data by about two dozen attendees inspired by Janice's thoughtful presentation certainly made me realize that our healthcare policy is like that drunk. Here is what I mean.

Our healthcare expenditures are completely devoid of any attempt at probabilistic thinking. I thought about the old Rand Corporation data, which I have presented here before. Juxtaposing them with the data on our National Healthcare Expenditures really drives home my message that we need to get a whole lot better at applying probabilities to our decisions. And this specifically applies to policy.

Just look at the glaring imbalance: while fully 60% of all premature deaths are due to behavioral, social and environmental factors which reside in the realm of public health, 97% of all NHE is spent on the medical side. If I add the 2% of the total NHE spent on research into the public health piece of the pie (this is exceedingly generous, as public health research gets a bafflingly tiny portion of the total US research budget), we still have 95% spent on personal health and its administration and only 5% on public health. 

So, if the probability of premature death due to a public health-related condition is 60%, why are we only spending 3% of all the healthcare dollars on fixing it? Another way of posing this question is, if the probability  of premature death from issues related to access to adequate medical care is 10%, why are we spending 97% of all the NHE on that piece of the pie?

If this isn't just like that joke, I don't know what is. Only in this case it is much less funny than in the case of the drunk.



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How medicine is like quantum physics

When a patients goes to his doctor to get fixed a pivotal triad of presentation-diagnosis-treatment ensues. The three steps are as follows:
1. History, physical examination and a differential diagnosis
When the patient shows up with a complaint, a constellation of symptoms and signs, a good clinician collects this information and funnels it through a mesh of possibilities, ruling certain conditions in and others out to derive the initial differential diagnosis.
2. Diagnostic testing
Having gone through the exercise in step 1, the practitioner then decides on appropriate diagnostic testing in order to narrow down further the possible reasons for the person's state.
3. Treatment
Finally, having reviewed all the data, the clinician makes the therapeutic choice.

These three steps seem dead simple, and we have all experienced them, either as patients or clinicians or both. Yet the cause for the current catastrophic state of our healthcare system lies within the brackets of each of these three little domains.

The cause is our failure to acknowledge the vast universe of uncertainty dotted sparsely with the galaxies of definiteness, all shrouded in false confidence. And while the cause and the way to address it are conceptually simple, the remedy is not easy to implement. But I am jumping ahead; first I have to convince you that I have indeed discovered the cause of this ruin.

Let's examine what goes on in step 1, the compilation of history and physical to generate a differential diagnosis. This is usually an implicit process that takes place mostly at a subconscious level, where the mind makes connections between the current patient and what the clinician has learned and experienced. What does that mean? It means that the clinician, within the constraints of time and the incredible shrinking appointment, has to listen, examine, elicit and put together all of the data in such a way as to cram them into a few little diagnostic boxes, many of which contain much more material than a human brain can hold all at once, even if that brain is at the right tail of human cognition (or not). What overtakes at this step is a bunch of heuristics and biases. Have we talked about those enough here? Just to review, heuristics are mental shortcuts that can serve us well, but can also lead us astray, particularly under conditions of extreme uncertainty, as in a healthcare encounter. If you want to learn more about this, read Kahneman, Slovic and Tversky's opus "Judgement under uncertainty: Heuristics and biases." As for cognitive biases, I will not belabor them, as there is enough material about them on this web site and elsewhere to overload a spaceship.

The picture that emerges at this step is one of fragments of information gathered being fit into fragments of studies and experience, stirred with mental shortcuts and poured into a bunch of baking tins shaped like specific diagnoses. Is there any room in this process for assigning objective probabilities to any of these events? Well, there is an illusion of doing so, but even this step is done by feel, rather than by computation. So while there is some awareness of a probabilistic hierarchy, it is more chaos than science. Given this picture, it's a wonder it actually works as well as it does, don't you think?

The next step in this recipe is the diagnostic workup. What ensues here is utter Wild West, particularly as new technologies are adopted at breakneck speed without any thought to the interpretation of data that they are capable of spitting out. Here the confusion of the first step gets magnified exponentially, just as it seduces us into further illusion of certainty. The uncertainties in arriving at the differential get multiplied by the imperfections of diagnostic tests to give the encounter truly quantum properties: you may know the results or you may know the patient, but you may not know both at the same time. What I mean is what I have always said on this blog: no test is perfect, and because of this simple truth, unless we know the pre-test probability of the disease in a particular patient, as well as the characteristics of the test, we have no idea about the context of these results. Taking them at face value, as we know, is a grave error.

What follows these results is frequently more diagnostic hit-or-misses, as the likelihood of harm and escalating expenditures without any added value rises. Then comes the treatment, with its many uncertainties and the potential for adverse events, and what are we left with? A pile of costly and deadly steaming manure. So, what's a doc to do?

I think that there is a very simple solution to this, and in its simplicity it will be incredibly hard to implement: education. And I don't just mean medical education. Everything that I have talked about in this post echoes back to the concept of probability. In the secondary education, at least as I remember it, probability is left to Advanced Math. By the time a student becomes eligible to take this course, she has been made to feel that she does not have the facility for math, and that, furthermore, math is boring and useless. So, while my friends in education may have a much better idea of what percentage of kids leave high school having been exposed to some probability, my guess is that it is woefully small. And those that do get exposure to it walk out of class perfectly able to bet on a game of craps or a horse race, but no clue how to apply these ideas to the world they live in.

And so those who progress into healthcare and those who don't have heard the word "probability," but cannot quite understand how it impacts them beyond their chance of winning the lottery. And unfortunately, I have to tell you that, if I relied on what I learned in medical school about probability, well, let's just say it is highly improbable that we would be having this discussion right now. This is why I do now and will for the foreseeable future harp on all of these probabilities, so that when you are faced with your own medical decisions, you will at least know the right questions to ask.

I know I need to wrap this up -- I saw that yawn! Here is the bottom line. First, we need to acknowledge the colossal uncertainties in medicine. Once we have done so, we need to understand that such uncertainties require a probabilistic approach in order to optimize care. Finally, such probabilistic approach has to be taught early and often. All of us, clinicians and patients alike, are responsible for creating this monster that we call healthcare in the 21st century. We will not train it to behave by adding more parts. The only way to train it is to train our brains to be much more critical and to engage in a conversation about probabilities. Without this shift a constructive change in how medicine is done in this country is, well, improbable.              

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Tinkering with health is not a laboratory job

Do you love Brussels sprouts? I do. And broccoli, chard and kale, too. Why do I ask?

Well, last week my friend Kent Bottles did a blog post on what the future of medicine may look like according to two of our prominent medical futurists, David Agus and Eric Topol. It left me scratching my head, so I went to find Agus on the interwebz, and came upon his 2011 TEDMED talk, which can be found here. The Brussels sprouts were just the beginning of nearly 20 minutes of bewilderment. I will get to the meat (ahem) of it momentarily, but I just could not get past his assertion at around 3:48, where he states that people in their 90s do not take up healthcare resources -- no mechanical ventilation, no weeks in the ICU -- and that they "die with dignity from whatever process ails them at that point." Really? In what country?

Here is the reality -- you can consult the Dartmouth Atlas for a lot of this info, but many other sources exist as well. There are approximately 2.5 million deaths in the US annually. Fully 1/3 of them occurs in the hospital, more that 1/2 of which involve ICU care. And incidentally, not to get all cost-conscious or anything, but 80% of all of the associated costs were due to ICU care. But wait, you say, this is not necessarily people in their 90s, right? OK, let's take it down a layer.

Among the 1/3 of all the annual deaths that occur in the hospital, nearly 3/4 are among the Medicare population, or those who are 65 years old or older. Furthermore, according to none other than the Dartmouth Atlas, up to 1/4 of all Medicare enrollees spend 1 week in the ICU in the last 6 months of life. OK, then. So, where are the data that old age is associated with low medical costs? Not here, that's for sure.

After this dubious beginning, Dr. Agus states the undeniable: humans are complex systems, and we need to think of them as such. Additionally, he advocates skepticism because much of what is done in medicine is not based on "true" data. OK, I can certainly go along with that. Then he goes astray. Here is how.

At around 10:30 he gets into technological solutions. You may be surprised that I do not fundamentally disagree with technology as the answer to disease. No, I disagree that technology is the answer to health -- this is where Brussels sprouts come in. At about 11:00 he starts to talk about aspirin and all the fantastic health benefits that are associated with it -- here is a screen shot of his slide:

And he suggests that aspirin should be mandatory, and that society should not have to pay for these diseases that develop due to what? Aspirin deficiency? Now, as veteran readers of my blog, do you see something funny about this slide? Is there something missing? Yes, you are right, where are the references for these statements? No, I did not cut off the bottom -- there are no studies referenced. One other critical piece of information is missing: whenever data on benefit are presented, data on risks must also be presented. Where are they? So, yes, be very skeptical. One more picky point: he brings up Michael Dell at 11:30 or so, telling the story of how his employees who smoked had to pay higher insurance premiums. By extension, Dr. Agus contends that we should charge higher premiums to employees who do not comply with aspirin. Unfortunately to get into the full controversy about the role of aspirin in these conditions is way beyond the scope of this post. But do let me give you a taste of what a balanced discussion of aspirin as a prevention for heart disease looks like -- here the Mayo Clinic web site is exemplary in providing a well-informed approach. Around 12:00 Agus builds the same argument for statins. And then he knocks down vitamins and supplements and suggests that people who take them should be penalized with higher premiums. OK, in my humble opinion this branch of inquiry has always been a fool's errand in a society that is fairly well fed, but higher premiums? Come on. I will not belabor this. And finally he sprinkles his comments with a few words on the microbiome. 

So what does Dr. Agus seem to say overall? My impression is that he thinks that we should tinker with maintaining our health by looking to manufactured drugs, such as aspirin and statins, as well as whatever we learn from the microbiome (more drugs?). What is missing here is the discussion of the risks vs. the benefits of such tinkering in healthy people. What is also missing is data to back up some of his fundamental assertions (see above). 

So, final words? Technology is not the enemy. If used correctly it can help us understand and cure disease. Tinkering with the healthy human is the job of evolution, not the laboratory. The potential "unintended consequences" of such tinkering are too colossal to ignore.  

          

    

Is this double dipping? A new bipartisan House bill on oncology reimbursements

Here is another gem from the House of Representatives: a bipartisan bill to increase Medicare reimbursements to community oncology practices. While at first glance this seems like a reasonable idea, this detail is puzzling:

The so-called "prompt pay" legislation excludes certain discounts extended to wholesalers when calculating Medicare reimbursements and is strongly supported by oncologists.

Confused? Met too. Here is how I understand it. Many community oncology practices have set up infusion clinics, where they administer intravenous chemotherapy on site to their patients. To stock these infusion centers they deal with drug manufacturers and distributors to purchase the drugs at wholesale prices. The bigger the buy, the bigger the manufacturer discount. To the best of my knowledge these discounts are proprietary information, guarded like state secrets. Yet despite these discounts, the clinics charge Medicare a premium for the drugs themselves as well as for the service of administering. The way this legislation looks to me is that it will completely eliminate any reduction in reimbursement related to these discounts. Double dipping, anyone?

Now, I have many friends who are oncologists, and this is really not a slur against them. But these infusion clinics have always represented a cash cow for these practices. And who would not want to have a steady source of income to maintain a robust practice and have some money left over for a life? Again, this is not an indictment of community oncology practices. If, however, one takes an external perspective, this bill becomes something of an anathema to improving efficiency of healthcare delivery. If the reimbursement rates for administering these already exorbitantly expensive drugs improve further, will it not become even more difficult for an oncologist to tread the fine line of the conflict of interest between treatment only when it is in the patient's best interest and treatment for income optimization? Again, I want to point out that I am not singling out oncologists, as it is a part of the human condition to rationalize our selfish decisions by putting them in an altruistic light. And given the amount of uncertainty about who might respond to these drugs, it is easy to convince oneself that a trial of a therapy may be a reasonable idea, with the reimbursements providing a nudge in that direction.

A couple of quotes from the sponsors of the legislation are also worth reprinting:

"On any legislation today, you have to find a way to pay for it. And like any legislation, that's an issue with this one," Whitefield said. 

"But to be truthful, because of the oncologist groups and patient groups and others, we think that there may be some provisions in the healthcare bill that passed last year that we may be able to utilize some of those funds for this. All of it's about healthcare, and if we can convince people that this is more important than the others then we can do it."     

On any legislation today? You mean it has not always been like this? I guess we have all gotten used to credit as a life style, and now it is time to pay the piper.

Now, what about this: "Because of the oncologist groups and patients groups and others..."? Are they saying what I think they are saying? That because groups are likely to benefit are saying that this is vital, it is in fact vital? I also have to wonder who those "others" are. Hmmm, I wonder...

And this: "All of it's about healthcare, and if we can convince people that this is more important than others then we can do this." OK, so the statement is so grammatically abominable and non-sensical that I can interpret it any way I like. And it seems to me that they are implying that increasing these already hefty reimbursements is more important than stuff like paying for prenatal care and immunizations to the poor? And other essential services to the Medicare population? Well, if this is not the a poster child for why we need to be articulating the value of healthcare, I do not know what is.  

New treatments: What benefits at what costs

Yesterday brought quite a bit of press coverage to a small biotechnology company in Cambridge called Vertex. All this attention was spurned by their gene therapy trial results in cystic fibrosis. The treatment, aimed at a genetic mutation present in about 4% of all CF sufferers, was able to improve the volume that a patient can force out of his lungs in 1 second by over 10%, from about 65% to 75%. Matthew Herper of Forbes on his blog, while being duly impressed by the results, also cautioned that the annual price tag for this medicine is likely to reach $250,000 per patient. So, what does all of this mean in the context of our ongoing national discussion about the value of therapies? Well, let's break things down a bit.

First, let's talk about CF. This is a genetic disorder that essentially makes mucus very sticky. Among its many effects, in its most familiar manifestation this mucus plugs up the airways making it difficult to breathe and predisposing the person to frequent and serious lung infections. When I was a resident back in the early '90s, I remember a devastating case of a young man in his late teens with CF whom we all knew so well from his frequent admissions for exacerbations. Though he was pretty high on the lung transplant list, he ended up succumbing to a devastating pneumonia in our ICU, leaving behind a devoted sister who had been fortunate enough to benefit from a transplant several years earlier. This was a typical course in those days: a brief life punctuated by frequent exacerbations, hospitalizations, antibiotics, gastrointestinal complications, and early death in the second or at best third decade of life with very little hope of procreation. Over the last 20 years things have changed dramatically in the treatment of CF: fewer exacerbations, much lengthened life expectancy and a good chance of having children. Yet we cannot attribute most of these changes to dramatic new breakthrough therapies. To be sure, while there have been tweaks to how we give antibiotics and how pancreatic enzymes are administered to replace the digestive enzymes that the pancreas in CF is unable to produce, most of the progress can be attributed to the increased attention to detail and the advent of almost ruthless care coordination at specialty centers. As a Fellow in the '90s I participated in a clinic where CF patients were transitioning from care by pediatric Pulmonologists to that by adult doctors. The CF specialist running this clinic did not only know all of his patients and their family members by names, but was available 24/7 to them and to his staff for consultation. This is the kind of dedication and vigilance necessary to improve the outcomes in CF.

Now, let's talk about the lesion addressed in the Vertex trial. The type of chronic lung disease caused by CF is called "obstructive." Simply put, it makes exhaling the air in the lungs difficult to do. On lung testing one manifestation of obstruction is the amount of air one is able to force out of his lungs in the first second of the effort, and this is called the FEV1, or forced expiratory volume in 1 second. Another important measure of the degree of obstruction is the amount of air that this volume expired in 1 second represents as a proportion of all of the air in the lungs that can be expired, known as the FVC or forced vital capacity. We say that if the FEV1/FVC ratio is under 75%, then obstruction is present. The size of FEV1 helps us understand how bad the obstruction is.

With this as a background, the primary outcome in many obstructive lung disease trials is the improvement in the FEV1. In the specific trial discussed, the average starting FEV1 in the intervention group was about 65%, which falls in the mild-to-moderate category of obstruction. What this means in terms of symptoms can vary widely. The 10% absolute improvement seen in the intervention group resulted in the average FEV1 of about 75% after treatment, definitely representing fairly mild obstruction (generally FEV1 over 80% is considered to be in the normal range). And this truly is impressive. However, equally interesting is the information that is not in the press coverage, largely based on press releases and sound bites from company executives, since the peer reviewed study is not available at this point. We are not told, but led to assume that, the control group started out on average with a similar deficit in lung function. We are informed that the treatment patients were 55% less likely than placebo patients to have an exacerbation of their disease, yet we do not know what the absolute numbers are; that is we are not told what proportion in each group had an exacerbation, how frequently or how severely. So, this 55%, in the absence of context, while an attention grabber, is not a substantive number. Herper does tell us that there was a remarkable difference in the weight gain (a desirable outcome in the CF population), on average 6.8 lb in the treatment vs. 0.9 lb in the placebo group. This is truly impressive, though it would be even more so if I knew that the trial was double blind, a piece of information I did not notice in any of the reports. Some of the reports have also alluded to symptomatic improvement in shortness of breath, though nowhere did I see this quantified.

The most important piece of data, however, is conspicuously absent from all the stories. What is the proportion of patients who responded to therapy? Why is this important? Well, we know that far from everyone responds to every treatment that they ostensibly qualify for; this is referred to as the heterogeneous treatment effect, or HTE. It is very likely that the 10% improvement in the FEV1 represents at once an inflated estimate referent to those non- or under-responders and a muted one for those patients with a terrific response. The question of a minimal clinically significant change in the FEV1 has haunted the lung trials community for a long time now. Yet, without setting some threshold for a minimum FEV1 improvement that correlates with a meaningful improvement in symptoms, one cannot quantify how well the drug works and hence articulate its value. This is crucial when trying to justify the ostensibly exorbitant price tag anticipated for this drug. How many patients will we need to treat in order to have one of them respond meaningfully with an improvement not just in a laboratory number, but also in their lives? If this targeted drug produces a desirable response even in 50% of all patients with the specific mutation it targets, then it means that we need to spend $500,000 annually to obtain a meaningful improvement in symptoms in one CF patient. But what if it only works this way in 20%? Then we will need to treat 5 patients with this drug to obtain 1 meaningful response at the price of $1.25 million annually. This becomes a bit more daunting, particularly given that the costs will have to be covered through some kind of public or pooled funds and given that this is one of many therapies in the pipeline likely to come with a similar conundrum.

I am not implying that improving a single life is not worth $1.25 million annually. In fact, it may well be a bargain. My point is that these are the serious discussions we need to have as a society, so that when the time comes to make these choices, the discussion will not be subverted by a few loud voices sensationalizing "death panel" slogans. Manufacturers need to know that they should disclose full data, not just selective tidbits that highlight benefits only, but also those difficult pieces of information that shed light on their costs. On our part, we need to understand the gargantuan effort and resources these companies expend to tame these elusive wild therapies that hold so much more promise in the abstract than they end up embodying.

We tread a fine line here. Information and how we assimilate it are the next frontier for cogent decision making. We need to get educated about this now because this train is leaving the station regardless of how we feel about it.                            

What's in a name or the furor over the proposed PA name change

Much like many other news items, I came upon the one about the proposed name change for Physician Assistants quite by accident: it came to me as an e-mail notification of a new topic being discussed on one of the physician only discussion boards that I am a part of. Apparently, after 40+ years of the profession's existence, there is a grassroots effort afoot to upgrade the name, and presumably the clout, to Physician Associate.

Well, as you can imagine, while the move is met with praise by the PA profession, the MD profession is seething. Some of the comments that I have seen from my colleagues betray such tremendous pain and suffering as a profession that it threatens my equanimity: I feel organically how lost we are as a profession to be expressing such bile without much thought over what appears to be a relatively innocuous event. But surprised I am not, and here is why. The medical profession's victory over all other potential modalities is hard-won and filled with a history of major turf battles and occasional demagoguery. The historically either-or approach of modern-day practice of medicine is responsible for the current landscape of our healthcare. In short, physicians have been only too successful at becoming the final word in health, at the exclusion of all others. With the allied providers, such as nurse practitioners and PAs, gaining in importance, particularly at this time of great uncertainty about the future of our healthcare "system",  understandably the MDs are reflexively bracing themselves for any and all turf battles. So, the perception of a power grab that this proposed name change has engendered in my hallowed profession is a classic fight-or-flight response, an activation of the survival instinct.

There are several aspects of this response that I find disturbing. At the most basic level, the response betrays such tremendous emotional pain among so many good people that it is all I can do to keep myself from sinking into a depression. And while I feel compassion for them, I am also forced to remind them that, as Eleanor Roosevelt once said, "No one can make you feel inferior without your consent". Applying the thought to the current situation, how the society may view PAs, whether they are called assistants or associates, should have absolutely no bearing on how physicians are perceived. Simply put, this perceived elevation in the status of the PAs relative to that of the MDs should not in any way make the MD profession diminish in its or the public's view.

The next layer of why this is a dysfunctional response lies in a poor choice of battles that this represents. I once had a boss, whom, despite working for myself currently, I frequently allude to as "the best boss I have ever had". When I would get hot under the collar, she would pointedly ask me to clarify for myself whether this was an issue to fall on my dagger for, thus teaching me that falling on my dagger too many times would make me politically into Swiss cheese, or, worse yet, dead. Under the circumstances, do MDs and their organizations really feel that this is an important dagger to fall on? In the current atmosphere of public distrust rightly or wrongly bestowed upon the profession, such indiscriminate issue picking will rightfully appear self-serving.

Finally, for a profession with, on average, a very high intelligence quotient, I am amazed that we are focusing on the minutia instead of looking at the big picture. Healthcare is a behemoth, an inefficient and inequitable trough at which there has been a feeding frenzy for too long. We need to be reining it in to the best of our abilities. And yes, altruism, not unmitigated self-interest should be driving us to do this. Gentleness toward and respect for each other, our communities and our planet should be the values that determine our actions as a profession. I am convinced that these are the values that brought us into medicine.

These are difficult times, made more so by the external forces all ganging up to deprive us of our humanity. Let's get back to the reasons why we went into medicine; let's sit quietly and find that lost thread of contentment and pride. Or else, if there is no joy left for you in your practice, resolve to find something else that you can be happy about. And no, it is not easier said than done. It is much more difficult to go through life carrying the baggage of self-imposed misery than to set it down in favor of finding happiness in this brief sojourn that is our life.                

Republican healthcare paradox

Reading the transcript in the WSJ of the remarks at the President's Healthcare Summit by Paul Ryan, the ranking Republican on the House Budget Committee reminded me of an occasional altercation during my childhood. When a friend would break a favorite toy, the temptation was to ask for the impossible: I want it back exactly the way it was, right now! Over the years this kind of a debating style thankfully dwindled in favor of more reasonable and logical solutions. How is it that our political discourse has spiraled into the childish domain of the unreasonable?

Here is the disconnect: on the one hand, as Mr. Ryan points out, the Obama-supported healthcare bills may bend the cost curve in the wrong direction, increasing drastically the deficits and creating empty promises for the future generations. This is clearly undesirable. But where was Mr. Ryan when Sarah, Michelle and the tea baggers were crying "death panels" and government sanctioned euthanasia when the talk came around to curbing the potentially unnecessary and marginally effective care that ravages our healthcare system? How can we have it both ways? Is it not obvious that if tthe amount of healthcare delivery continues its upward trajectory, the bill accompanying it can do nothing but continue to go up as well?

The Republicans insist that they merely object to governmental intervention in the healthcare market, and they would be delighted to craft legislation that promotes free market solutions to this mammoth that is killing America. Really? When was the last time any congressional legislation promoting "free markets" helped the people who are struggling? If the fact that in 2004 the top 0.01% of the US population controlled 6% of the nation's wealth is any reflection of the success of such legislative efforts, well...

This spin machine has got to stop. We need to demand that our legislators speak honestly and rationally about this complex multifaceted issue. The reality is that all Americans need to do some soul-searching in order to make the adult decisions that need to be made. What are we willing to give up and what are the trade-offs we are willing to accept? As difficult and as unusual it is for us to think of giving something up, make no mistake about it: we must give some things up.

So, no more sound bites! Give us the respect of an informed and cordial debate without leaving out critical pieces. Stop running for office and govern!

Would you spend your entire paycheck on healthcare?

It is an absurd question, right? Well, not really. Bear with me for a bit.

As I half-listened to the White House healthcare summit yesterday, I was feeling the familiar sensation of nausea rising in response to the usual excuses and talking points from both sides. Talking at each other, parroting old memorized lines about the process rather than the substance, the illustrious group came away with no consensus. Nevertheless, this lack of results seems to be pushing the Democrats to plow through the opposition and unilaterally pass the legislation through the process of reconciliation. But will it be done well? This has always been the question.

I think this WSJ article from today has called our attention explicitly to something that has been hampering any sensible approach to the reform, the selfish blame game:

Insurers contend that they must pass on ever-higher bills from hospitals and doctors. Hospitals say they are struggling with more uninsured patients, demands by doctors for top salaries, and underpayments from Medicare and Medicaid.

And doctors say they are strong-armed by insurance monopolies and hampered by medical malpractice costs.

This is the ultimate case of missing the forest for the trees. Let's think about this in slightly different terms. You are noticing that your food bill is growing out of pace with your income, the growth of your family and the growth of the national inflation rates. Do you not stop and ask what is going on? And if the answer is that, without any other changes, your family is now consuming 20 pounds of potatoes per week, while 6 months ago you made do with 4 pounds, do you not stop and ask why? And if on top of that observation you have also noticed that your family is becoming obese, do you not stop and rethink what is going on? And if as you stop and think you notice that your obese family members are in fact making even more frequent trips to the kitchen to feed their increased hunger, do you not want to break the cycle?

Well, we are "eating" a lot more healthcare than we did 10 years ago, and in the next 10 years we are likely to be "eating"even more. If healthcare continue to grow at the current pace, we will be spending 100% of our income on healthcare before we know it; and on healthcare that continues to fall short of meeting our needs to boot. The levels of chronic disease in our society have never been higher, and the number of diseases that "require" treatment has never been more vast (think mild depression, erectile dysfunction, lactose intolerance, and many other marginal human woes, most best addressed by introspection and lifestyle modification). Reflecting this growth, our preoccupation with health and disease seems to be edging out other, far healthier pursuits. And the rhetoric of "best healthcare system in the world", emanating from the Republican opposition yesterday, is not only disingenuous, it is just stupid, especially coming from someone, in fact many someones, who should know better.

So, let's look at the big picture, folks. Let's walk away from the divisiveness of the blame game and get back to the basics. We are not the best, we are least accessible, and we are most expensive. In the world! Yes, we are special, we are Americans. But it is time to exercise that specialness by admitting the abject failure of this market experiment, get off our high horse of individuality and look to other nations and systems for a sensible solution. I for one am not willing to spend all of my time or money obsessing about my health. How about you?  

 

When the black box of evidence is empty

A close friend of mine gave birth a few days ago to her second child. My friend is a slight woman whose infant came out at 6 lb 9 oz, even though she was calculated to be at 36 weeks' gestation. The infant and the mom have done great with absolutely no complications.

This is why, when I was talking to the Dad this morning, I was shocked to hear that the baby had to undergo a test I had never heard of: something to do with putting the child in its car seat and monitoring its heart rate and oxygen saturation for one hour. Mind you, my youngest is 9 years old, and I had never hear of this test, so I looked it up.

Turns out, this is a test formally known as a "pre-discharge car seat challenge". I went to the American Academy of Pediatrics web site to look for their take on it, and here it is:

Hospitals should develop a policy to ensure provision of a period of observation in a car safety seat before hospital discharge for each infant born at <37 weeks' gestation to monitor for possible apnea, bradycardia, or oxygen desaturation.

I went on a circuitous journey to trace the origin of this recommendation. Turns out, it is based on a consensus statement from an APP committee from 1996, the reference for which is this paper from 1993 about implementation of the 1990 AAP recommendations. Looking at the 1990 version, however, does not help in the least, as there is no mention at all of the test in question, or anything to do with infants under 37 weeks' gestation. So, this is where the dubious evidence trail ends. Period. No evidence near as I can see. [If someone knows of a more complete statement that does include the information I am interested in, please, point me to it].

Why did I go after this piece of information? Well it seemed to me to arise out of the "more is better" fallacy, and I wanted to understand a) the evidence that led to the recommendation, b) how the test alters practice, and finally, and most importantly, c) does this practice impact the desired outcomes (i.e., prevent something bad).

So, I went to one of the most trusted sources in evidence synthesis, the Cochrane Collaborative, to see if they have examined this issue. Lo' and behold, I found this 2006 review, whose findings were summarized thusly:

There is no evidence that undertaking a pre-discharge "car seat challenge" benefits preterm infants. The "car seat challenge" assesses whether preterm infants who are ready for discharge home are prone to episodes of apnoea (stopping breathing), bradycardia (slow heart rate), or desaturation (low oxygen levels) when seated in their car seat. However, it is not clear whether the level of oxygen desaturation, apnoea, or bradycardia detected in the car seat challenge is actually harmful for preterm infants. Additionally there is concern that the use of the car seat challenge may cause undue parental anxiety about the safety of transporting their infant in a car seat. Despite these uncertainties, and despite the widespread use of the test, we have not identified any randomised controlled trials that assessed whether undertaking a car seat challenge is beneficial or harmful to preterm infants.

So, here is a kid, born at supposedly 36 weeks gestation, but looking and acting like a full-term infant, having to sit in her car seat for 1 hour to undergo monitoring that has absolutely no evidence behind it. Putting aside the inconvenience, the false positives, the parental anxiety, let's think about the costs. I could not find published reimbursement rates for this useless test. However, if you think about the person-power, the equipment used, and the potential prolongation of hospitalization that it may induce, the price tag is likely not trivial.

This case is instructive to me. As I have said before, a pitiful minority of medical practice has good evidence behind it. It is precisely this type of testing, done to comply with what looks to be a self-propagating yet outdated recommendation, that healthcare reformers in Washington need to be addressing. Furthermore, when evidence is kept in a black box, we are likely to find that the box is indeed empty.a,

bradycardia, or oxygen desaturation. An appropriate

hospital staff person should conduct the observation.

Hospitals should develop policies to

  

An executive articulates the value of his drug

Allos Therapeutics is a small biopharmaceutical company located in Westminster, Colorado, with a single agent on the market. Pralatrexate, brand name Folotyn, is a small molecule therapy for a rare and aggressive hematologic malignancy peripheral T-cell lymphoma. The recently FDA-approved drug is stirring controversy by, you guessed it, pricing itself out of the market. The pricing giants at the company decided that a fair price for the drug, achieving parity with other compounds in the space and helping them recoup their investment, would be $30,000 per month.

In the area of cancer, such a price tag is certainly nothing unusual. Here, fancy and expensive-to-produce biologic therapies can run as high as $100,000 annually to treat such common cancers as those of the lung, breast and colon, even while only prolonging the patient's life by an average of 2 months. But here is the kicker: pralatrexate is not a biologic, but a small molecule, and not even first in class! So, essentially, it is a me-too drug that is not particularly expensive to manufacture.

But let's give the company the benefit of the doubt -- after all, clinical development, especially in a rare cancer, is prolonged, costly and generally resource-intensive. A few more pieces of the puzzle are in order before we can make the final judgment. The drug was approved based on a trial of 115 patients with recurrent PTCL refractory to, on average, 3 prior therapies. The outcome evaluated was a combined endpoint of complete response or complete response unconfirmed or partial response (each indicating degrees of tumor shrinkage). Among the 111 evaluable patients the response rate was 27%, and the median duration of response was 9 months (meaning that one-half of the 29 responders progressed by 9 months). And the median duration of use of the product in the trial was 70 days.  

So, let's do the math here: if 100 patients are prescribed this drug for 70 days (this is being conservative, as the median is usually lower than the mean value in similar distributions) at a cost of $30K per month, we have spent $7,000,000 to get a response in 27 patients that lasts under 1 year, or about $333,000 per year of life saved. So, this may be less reasonable in some books than others. Hmmm...

Well, in case you you have any shred of doubt remaining, look at what James Caruso, the Chief Commercial Officer for the company, is quoted as saying:

Patients, moreover, are likely to use the drug for only a couple of months because the tumor worsens so quickly, he said. So the total cost of using Folotyn will be less than for many other drugs with lower monthly prices.

So, the message is that our expensive drug should be used not because it saves lives, not because it improves quality of life, but because the expense will be limited by the its uselessness? Isn't is a little bit like saying that a gas-guzzling HUM-V is worth the expense because it will break down in two months anyway? I have to admire the manufacturer in speaking the truth this way: if more manufacturers do this, comparative effectiveness concept will become obsolete before it is even legislatively approved.  

Can US agriculture reform inform the healthcare debate?

A rather shocking analysis by an otherwise astute observer of American history Athul Gawande, MD, in the New Yorker has sent me reeling from its short-sightedness. In his article Gawande argues that the Senate healthcare reform bill is on the right track by not demanding wholesale budget controls, but rather instituting small pilot programs to see what works best. Although pilot programs may not be a bad idea, Gawande's argument is deeply flawed. It relies on the theory that the changes in agriculture and food production in the beginning of the 20th century were also characterized by small pilot programs that eventually took over the nation, bringing our food budget under control and, by reducing drastically the proportion of population engaged in farming, provided a productive work force for manufacturing and growing the economy.

Indeed, the changes in the way we produce our food that took place in the first decades of the last century are remarkable, and one can stand breathless before this edifice of modern science with unabashed awe... but only if one truly believes that this movement has been a success. And clearly, Gawande subscribes to this opinion:

The history of American agriculture suggests that you can have transformation without a master plan...

I would beg to differ, and so would many who worry about our current agricultural practices. We raise corn and soy beans as monocultures, and douse them with petroleum-based fertilizer to make them grow. The humus that Gawande talks about in his article is not considered at all in today's industrial agriculture, and the level of the destruction of the topsoil in these gigantic swaths of potentially fertile land is unprecedented. Similarly, food animals are grown in inhumane, crowded, disease-laden conditions in so-called Concentrated Animal Feeding Operations, or CAFOs, where cows are fed corn and corn byproducts. Instead of grazing in pasture and putting their rich manure back as fertilizer, thus ensuring the sustainability of the humus, their effluent now contaminates water supplies. It was only a little over 20 years ago that the world was shocked by the revelation that these ruminants were in effect made into cannibals by being given parts of other cows in their feed, resulting in the emergence of the Mad Cow Disease. I doubt that I even need to mention that because of the unhealthy conditions of the CAFOs, animals are actually routinely given antibiotics to keep them from contracting infections, a practice that scientists agree is driving the evolution of a class of superbugs that are now threatening our species.

A couple of other effects of our wildly "successful" farming apparatus are the obesity epidemic, the combined contributions of deforestation for the sake of pasture and the exorbitant amount of methane gas produced on these farms to climate change. There are also the effects of the continuing fallout of the displacement of family farmers from their communities and their connection to ancestral ways to the impersonal and demoralizing widget-making in the cities. All in the name of the economy!

So, dear Dr. Gawande, forgive me if I disagree with you on each and every point about how industrialization of agriculture has been a wonderful thing for America. I believe you, much like most pundits, are confusing the almighty American Economy with the American citizens, who certainly fade in comparison to the riches derived on their backs.

I am not fundamentally opposed to pilot programs -- they may be the way to tweak incremental changes in how we do medicine. But to rely on them as the sole fundamental tool in the reform is a big mistake. In fact, the history of the American agricultural revolution is the strongest argument there is against a transformation without a master plan. It would be tragic to be lulled into the complacency of "act now think later" at this critical cross-roads of our time.        

When an information disconnect does not create cognitive dissonance

Just posted this on the BMJ's doc2doc blog  

Is it rare for an information disconnect to be accompanied by a complete absence of cognitive dissonance? And does such an absence signal mere ignorance or deliberate intellectual dishonesty? There is such a gap present in our healthcare debate.

Think about this. The Republican Party has always touted itself as a fiscally responsible party. Under this banner they like to cut taxes and minimize what they call "entitlement" programs (these of course are just the programs that provide a modicum of support for those left out of our consumerist equation). So, given this as a top priority in the hierarchy of their agenda, there is no contradiction in their stance against public coverage of healthcare in the US. OK so far?

Well, here comes the disconnect (no pun intended): they do not wish to limit any and all existing and future healthcare interventions for our public, including the Medicare population. They have made up rows of dying grandmas whose ventilator access is rationed by the government death panels, and have equated the proponents of rational rationing with the Nazis. So, where is the disconnect? Well, given that Medicare expenditures have been growing at rates far outpacing the US annual inflation (up to 12% in some years), how do we propose to pay for this escalation if we are unwilling to limit care? Well, one way is by raising taxes. This thought, of course, sends the Republicans sobbing into their hankies -- such a solution is anathema to their pseudo-science of supply-side economics. In the meantime, a few years ago they added to the Medicare bill by creating Medicare Part D, or prescription drug coverage. In and of itself, this coverage is a good idea. Unfortunately, Medicare is not empowered to deny coverage based on cost or cost-effectiveness, and it is further prohibited from negotiating drug prices. Well, in 2006 this program cost Medicare over $40 billion. And George W. Bush insisted on lowering taxes at the same time. And he started two expensive wars, too. So, am I just dense because I cannot get these numbers to add up? Am I missing something that is indeed fiscally responsible about the Republican stance? Why does this disconnect not create any visible cognitive dissonance for them?

I think I know why. They are not the party of fiscal responsibility, but the party of survival of the fittest consumer. An entitlement program creating corporate profit is good, while one geared at improving our society is a manifestation of the Nanny state. In fact, the Nanny state has run rampant in the last 30 years wiping noses of our corporate giants with their supply-side religion, while shaking off the needs of the public as socialist propaganda. There is no fiscal responsibility here, only the single-minded pursuit of more for less. The US currently has a $12 trillion (this is worth writing out just for its shock value: $12,000,000,000,000) debt on the books. This does not take into account what is already committed to future debt, which by some accounts exceeds utterly unimaginable $60 trillion. In fact, by 2019 the US government's annual interest on its debt will be $500 billion more than it is today, going from $200 to $700 billion! Just the interest, folks!

So, while I can almost intellectualize the Republicans' selfish desire to keep 46 million people in the US from availing themselves of healthcare, I deplore their dishonesty in hiding behind the aegis of fiscal responsibility. The last time our budget was balanced, a Democrat was in the White House. A Republican started an ill-conceived war and cut taxes at the same time -- this is living in a fantasy of a most destructive kind! So, don't make the healthcare bill about anything other than what it is about: the potential profit to be made from keeping healthcare status quo on the backs of growing numbers of citizens of our nation. It makes you look not only hypocritical, but also supremely stupid!                  

Is the art of medicine inefficient?

I was listening to NPR's Marketplace Morning Report today and heard that Hershey has an eye on the British confectioner Cadbury. Now, I love Cadbury Crunchies, the ones that you can only get in the UK, and dread the prospect of having them disappear. But that is off topic. The story talked about how the company, Cadbury, has become more efficient since it hired an American CEO, and it is looking to further those efficiencies. In this context the word "efficiency" was kind of like fingernails on a blackboard. Here is why.

When we talk about efficiency in the business world, we are basically talking about getting as much profit as possible. This profit is wrung out of the system by reducing production costs to the maximum extent possible and by charging the top price that the market allows. Some of the ways in which the US companies have increased their efficiencies over the last 30 years are 1). by moving manufacturing to developing nations, where labor is extremely cheap, 2). by reducing the US workforce to the bare minimum through increased use of automation (and don't we all look forward to talking to a computer when we are looking for customer service on the phone!), and 3). keeping down the US workers' wages at their 1975 levels, even as the productivity has grown exponentially.

So, now I come to my concerns about healthcare. Efficiency is one of the domains identified originally in the Institute of Medicine's report "Crossing the Quality Chasm" as a measure of a functional healthcare system. Since then, the Commonwealth Fund has consistently given a poor grade for efficiency in their annual report card. And there is no question that the system as it stands today breeds inefficiency.

On the other hand, I worry that in our traditional American single-minded zeal we will go overboard on efficiency in healthcare purely in the business sense. The Six Sigma models and similar lean techniques are designed for the world of business. Medicine, I would argue, is a densely cognitive field, and despite the illusion that computerization will obviate the need for human attention, we should always demand that a human being, not a computer, is thinking about our medical picture in a holistic way in our hour of need. So, while we really do need to get rid of the considerable amount of blubber in the system as it exists today, we should never tolerate the adoption of the the traditional business view of efficiency. We must be vigilant against reproducing the curve above in our healthcare system. And as much as health IT is seen as the holy grail of medicine, let us not work under the woeful misapprehension that this valuable and necessary tool can replace medicine's practitioners, who spend their careers cultivating the art of medicine, as well as the science. And if you do not believe that there is art to medicine, you have never had a serious encounter with it either as a clinician or as a patient.

The simple math of healthcare access

Look, here is the bottom line in broad strokes.

The costs of healthcare have been rising exponentially, but people's incomes have not. Despite the biggest economic expansion over the last 50 years, and despite astronomic rise in our productivity, the real wages for the bottom 80% of all earners have not increased one iota since 1975! This means that, while the costs for all products and services have grown at the pace of inflation or more rapidly (as in the case of healthcare), our buying power has remained stagnant.

What is the result of this disparity? Well, one result was the implosion of the mortgage-backed derivatives market. Some economists posit that the productivity-wages gap allowed corporations to stockpile enormous wealth. This wealth, in turn, was translated by the Wall Street Wizards into the obscure mortgage-backed derivatives Ponzi scheme, where mortgage loans lent to people with no way to pay them back were being used as collateral for these assets. The math is simple: productivity was up, wages stagnant, consumerism rampant, cash abundant -- bingo! The nation lived beyond its means for over a decade, imaginary wealth made and lost in a blink of an eye.

What does any of this have to do with healthcare? The connection is pretty obvious to me: rapidly escalating costs in the face of stagnant wages and diminished capital. Without any changes in the trajectory of the healthcare costs even more people will be unable to afford health coverage. This simple arithmetic should not be so difficult to grasp. Closer to home, anyone who now says "Not my problem, I can still get 'everything' ", prepare for it to become your problem. Who will pay for "everything"? Without the needed cost containment, the faces of those left behind by our cruelly inequitable system will be getting more and more familiar.

Will more healthcare bring better health?

An awful lot of time and energy is being spent on finding solutions to our broken healthcare system. This  is justified, of course, given the unprecedented 16.2% of the GDP that it eats up, while virtually excluding 46 million people from participating through lack of insurance, and harming many through poor and defensive practices. But will focusing on these unquestionably important trees improve the forest of the nation's health? A nation that boasts unprecedented rates of obesity, diabetes and other chronic diseases? A nation 40% of whose citizens suffer from a chronic disease? A nation where 80% of the adult population pops at least one pill every day?

I am skeptical. And here is why. The Rand Corporation created this very interesting pie chart, quantifying the influences of factors responsible for premature death in the US.

Based on this chart, only 10% of all premature deaths will be avoided if we fix the access to and quality of our healthcare system. If we posit that avoiding premature death is our primary goal, then perhaps we need to be exploring other risks that compete with the medical care short falls. This scheme suggests that of the 70% of the causes that are modifiable (we cannot change our genes yet), 86% can be attributed to our social status, the environment around us and our behavioral patterns. Let's examine each one separately and see what conclusions we might draw.

Socioeconomic status (SES) is known to be the single most important determinant of health. SES determines not only where we live, but also what we eat, how much activity we engage in, and our educational opportunities. It is well appreciated that obesity rates are higher in poorer communities. Most shocking, however, was a report a little over a year ago indicating that, in the face of ever-increasing life expectancy in the overall population, life expectancy in the poorest parts of the US is declining. At the same time, the economic gap in the US is widening, with an unprecedented 6% of the wealth concentrated in the top 0.01% of the population. Further, since the mid-1970s, the real wages for the bottom 80% of earners have remained constant, while productivity has enjoyed exponential growth. And are we doing anything about this driver of health? We are continuing to outsource jobs, we continue to tolerate astronomical bonuses on Wall Street, all with the blind belief in economic growth, but in reality at great expense to the majority's welfare.

Environment is next. We have not only passed Hubbert's peak in oil consumption, but we have gone beyond the level of CO2 production, 350 parts per million, beyond which we threaten the planet with temperature increases that will not only alter our geopolitical balance, but also potentially bring us to the brink of extinction. Yet, much like the tobacco "debate" played out in the decades of the 20th century, we continue to drag our feet on climate change. Furthermore, we use tobacco tactics to cast doubt on the potential links between other man-made environmental contaminants and disease, confusing absence of evidence with evidence of absence of such a causal link.

And what about behavior? In the 21st century we are not talking about sex and substance abuse. In the 21st century the addiction of choice is the media. Children spend on average 7 hours per day glued to a screen, being unabashedly marketed to even while in school. Three major corporate conglomerates control the content of TV programming, telling stories not in order to promote responsible citizenship, but so as to create the perception of need where none exists, to drive consumerism and finally to generate the next generation of corporate drones with the single aim of ever-increasing productivity.

If it seems that we live in the Brave New World, it is because we do. The healthcare reform discussion, though necessary, is taking a lot of air away from other, equally as or potentially even more urgent concerns. The lather stirred by the opposition to healthcare reform is an effort to imbue this almost no-brainer populist need with a false controversy in the hopes that the hot air will distract the already apathetic nation from the worsening crimes of Wall Street against Main Street.

So, no, I do not believe that solving our healthcare problem will resolve out health disaster. The latter will need a more global approach to our runaway societal woes. A few posts ago I indicated that the market model of healthcare was a miserable failure. Today I have to conclude that unopposed market forces have promoted the failure of many, the success of a few, and have sold our Democracy to the highest bidder. Only by injecting humanist values into our society can we hope to get this ship back on course. If we don't, we are simply enjoying our final days of debauchery on board of this titanic.

The cost-effectiveness of patient empowerment

I think by now there is not a person in the US who cannot quote at least approximately how much we spend annually on healthcare. Fewer people appreciate that nearly 1/3 of this $2.2 trillion bill is eaten by hospitalizations, amounting to about $680 billion. Although the data in the diagram below represent a single year, the overall distribution of expenses is remarkably constant over time.

Of this staggering amount of money, no less staggering is the ICU expenditure, quantified at nearly $60 billion in 2000.

The role of the ICU is to support a patient through his/her critical illness. The reason ICUs exist is that these patients require more intense human and technological interventions, and this geographic segregation allows for more efficiently concentrated care. The philosophy behind putting a patients in the ICU is that of a therapeutic trial. In other words, no one knows for sure a priori whether the ICU intervention will make any difference in the individual patient's outcome. What we have to go by is evidence from studies of similar patients that tells us what on average the expected outcome may be.

Until about a decade ago we never had the luxury of inquiring about what an ICU survivor might be doing a year later: we were fixated only on getting the patient through the acute illness. In fact, about 10 years ago, when I had the chance to ask a very well known and respected academic intensivist whether he cared about what happened to the patient once the ICU doors closed behind him, he, like so many of his peers, gave a resounding "no". Well, we have learned a lot over the last two decades about how to improve ICU outcomes -- more patients are surviving to leave the ICU. So now we have started to be concerned about their longer-term outcomes. After all, surviving an ordeal today just to die or, even worse, wish you were dead, tomorrow is not the kind of a victory anyone would want to claim. Unfortunately, what we are learning is that their long-term outcomes are not particularly encouraging. More than half of those patients who survive a prolonged critical illness die within the subsequent year. Even more discouraging is that fewer than 10% are actually at home living independently. So, when starting a therapeutic trial of an ICU, both the clinician and the family of the patient need to have a clear end in mind, so as to minimize pain and suffering for both, the patient and the family. And a byproduct of this conscious minimization of suffering through inappropriate care is potential avoidance of economic ruin.

What triggered this rumination for me is the paper published in this week's New England Journal of Medicine looking at the clinical course and outcomes of nursing home residents with advanced dementia.
In this prospective study following 323 patients, more than one-half were dead by 18 months. This is not in and of itself surprising. What should, however, shock an uninitiated reader is that 4 out of 10 of these unfortunate patients undergo at least one burdensome intervention, including a hospitalization or an ED visit, in the last 3 months of life. Although as a former clinician I have no trouble believing this number, as a member of the human species I am absolutely appalled! Is there really ever a point to such torment knowing that any potential postponement of death is at best temporary and at worst painful for the patient?

Another interesting point in this study is that what reduced the likelihood of this infliction of pain was a clear understanding by the patients' loved ones of their dismal prognosis. So, although some of the less informed yet loud and disingenuous voices tout them as government-sponsored death panels, the reality is that end-of-life discussions are not intended to limit necessary care. Rather, their intent is to create an honest and transparent dialogue between the clinicians and the patient and his/her family, thus empowering them to make the right choices according to their values. Perhaps the fear-mongers in their torrent of feverish activity have been too busy to notice that the age of paternalistic medicine is over. In the 21st century patient empowerment is the mantra. End of life discussions are just that, empowering.

If I were a politician driving a message, I would stop here. I would not connect this message of empowerment to dollars. But I am an outcomes researcher, so I must. There are examples in corporate America who say that if you do the right thing for the customer, the money will follow. To me this is simply an extension of the golden rule. Call it karma, call it what you will, but doing what is right is often contagious and causes a domino effect. And yes, in the case of spending the necessary time with the patient and the family to discuss the best course of action, the desired byproduct may be to help curb the waste of Medicare dollars on useless interventions, thus ensuring not only the best for the patient, but also the program's sustainability.

Healthcare reform: so much more than costs

The opposition to healthcare reform is fanning fears of rationing as a way of controlling escalating costs and broadening access. A better use of our collective energies would be to identify and eliminate the waste and harm produced by the current healthcare system. We are well aware of over 1 million hospitalizations and the nearly 100,000 deaths associated with hospital-acquired infections, at a cost of approximately $40 billion annually. We have this information partly because the data infrastructure within hospitals exists to track these events. Many more potential adverse consequences of healthcare remain unrecognized and unquantified, particularly those that occur outside of hospitals, where the majority of healthcare takes place. We hear distant echoes of alarms sounded by researchers, but because there is no integrated picture of the full magnitude of the harm, we remain complacent. We must admit that harm exists, develop systems to track and quantify it, and finally eliminate it. The time for this is now; here is why.

Why would harm be pervasive in what has been called by some “the best healthcare system in the world”? Theoretically, risk aversion, by promoting a culture of over-testing, over-diagnosis and over-treatment, is fraught with paradoxically noxious potential. Beyond theory, an example of how the “better-safe-than-sorry” approach can be detrimental plays out in screening mammography. Despite the fact that the US Preventive Services Task Force says to commence breast cancer screening at the age of 40, the risk and benefit balance of this recommendation is unclear. A recent study from Europe, for example, noted that for every 2,000 women screened over a 10-year period, one cancer death is averted. The trade-off is that 2-10 other women receive invasive and toxic treatments for a cancer that would not have become life-threatening. What is even more disturbing is that we do not know how many of these 10 women are consigned to life-long illnesses or an early death due to the complications of this unnecessary treatment. Similar concerns exist for prostate cancer screening and for many other diagnostic and therapeutic modalities we employ every day: the drug and device approval process focuses more on short-term benefits and less on long-term risks. Therefore, the full extent of the harm remains unknown.

Until recently, the heretical talk of harmful effects of healthcare was relegated to the fringes of the medical world. Fortunately, this conversation has now penetrated into the mainstream, with a paper in a recent issue of The New England Journal of Medicine laying out the research framework to explore this problem. Unfortunately, the research agenda, which still requires much clarification and planning, though harnessing the will to fill this knowledge gap, will be hard-pressed to find a way. This is because of the heterogeneous and still mostly paper-based medical record-keeping by the majority of US practitioners, precluding meaningful recognition and aggregation of relevant data.

This lack of knowledge gives the public a rosy view of healthcare: high benefits with virtually no harms. Getting a handle on the full picture of the balance of benefits with harms and waste, however, is critical for optimizing outcomes. Although data to quantify it are presently scant, the timing of the emergence of this research agenda, coincident with the major push to adopt electronic health data platforms along with the recent allocation of funding to the comparative effectiveness research (CER), is fortuitous. Electronic data, if captured accurately and uniformly, are a robust source of complete information on long-term outcomes, and the well-established framework for CER is a natural fit for incorporating these data to inform policy decisions.

If healthcare reform is to achieve its goals of universal access to good quality healthcare at a reasonable price, we must not miss this opportunity to align the research into harm with our ability to generate useful data from electronic sources to feed smart policy decisions through CER. Although historically our healthcare system has enjoyed very little integrated planning, this is an opportunity to draw up a sensible blueprint for a successful future. If we do it right, we will be not only saving money, but also addressing such an important societal concern as human lives.